2024 Approvals

Advancing Care for Chronic Graft-Versus-Host Disease with Niktimvo

Jasmine C

25 Nov 2025 — 1 min read

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What is the name of the drug and what does it do?

Niktimvo (pronounced nik-TIM-voh), generic name axatilimab-csfr, is a prescription medicine used to treat chronic graft-versus-host disease (cGVHD). It is approved for adults and children weighing at least 40 kilograms (88.2 pounds) after at least two prior treatments for cGVHD have not worked. cGVHD is a condition that occurs when transplanted cells attack the recipient's tissues. It was approved on August 14, 2024.

How does it work?

Niktimvo is a monoclonal antibody that blocks the colony-stimulating factor-1 receptor (CSF-1R). This receptor is found on certain immune cells, including macrophages. By blocking CSF-1R, Niktimvo reduces inflammation and fibrosis (scarring) caused by overactive immune responses in cGVHD.

What did the research discover?

In clinical trials, Niktimvo showed promising results in treating cGVHD. Among patients who had tried multiple other treatments without success, 75% experienced improvement (partial response). The median time to see results was about 1.5 months, with some patients maintaining their improvement for a year or longer.

What are some of the side effects?

The most common side effects include:

Infections
Increased liver enzyme levels (AST and ALT)
Nausea
Headache
Low energy
Diarrhea
Muscle, joint, or bone pain
Low blood cell counts (anemia)
Increased levels of calcium or certain enzymes in the blood

Serious infusion-related reactions, such as fever, chills, or difficulty breathing, can also occur. Your healthcare provider may give medications like antihistamines or acetaminophen before each infusion to prevent these reactions.

What are the dosage recommendations and how is it prescribed?

Niktimvo is given as an intravenous (IV) infusion over 30 minutes every two weeks. The dosage is based on body weight, with a maximum dose of 35 mg. A healthcare provider administers the medication, and the number of infusions depends on the patient's response and side effects. Blood tests are done regularly to monitor for adverse effects.

Source:

U.S. Food and Drug Administration. Full prescribing information: NIKTIMVO (axatilimab-csfr) injection. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761390s000lbl.pdf. Accessed 2024 Nov 21.

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